Stem Cell Transplantation With NiCord® (Omidubicel) vs Standard UCB in Patients With Leukemia, Lymphoma, and MDS
- Conditions
- Hematological MalignanciesAcute Lymphoblastic Leukemia (ALL)Acute Myelogenous Leukemia (AML)Myelodysplastic Syndrome (MDS)LymphomaChronic Myelogenous Leukemia (CML)Acute Leukemia
- Interventions
- Drug: NiCord® (omidubicel)Other: Cord Blood Unit
- Registration Number
- NCT02730299
- Lead Sponsor
- Gamida Cell ltd
- Brief Summary
This study is an open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord® versus transplantation of one or two unmanipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome, chronic myeloid leukemia or lymphoma, all with required disease features rendering them eligible for allogeneic transplantation.
- Detailed Description
Successful blood and marrow transplantation (BMT) requires the infusion of a sufficient number of hematopoietic stem/progenitor cells (HSPCs), capable of both homing to the bone marrow and regenerating a full array of hematopoietic cell lineages with early and late repopulating ability in a timely fashion.
A major drawback of Umbilical Cord Blood (UCB) is the low stem cell dose available for transplantation, compared to mobilized peripheral blood (PB) or bone marrow. This low stem cell dose can compromise the chances of engraftment and contributes to delayed kinetics of neutrophil and platelet recovery, as well as other transplant outcomes.
The aim of ex vivo expansion of cord blood is to provide a graft with sufficient numbers of cells that have rapid and robust in vivo neutrophil and platelet producing potential to enable successful transplantation.
NiCord® is a stem/progenitor cell-based product composed of ex vivo expanded allogeneic cells from one entire unit of UCB. NiCord® utilizes the small molecule nicotinamide (NAM), as an epigenetic approach to inhibit differentiation and to increase the migration, bone marrow (BM) homing and engraftment efficiency of Hematopoietic Progenitor Cells (HPC) expanded in ex vivo cultures. The chief aim of the study is to compare the safety and efficacy of NiCord® single ex-vivo expanded cord blood unit transplantation to unmanipulated cord blood unit transplantation in patients with hematological malignancies following conditioning therapy.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 125
- Applicable disease criteria
- Patients must have one or two partially HLA-matched CBUs
- Back-up stem cell source
- Adequate Karnofsky/Lansky Performance score
- Sufficient physiological reserves
- Signed written informed consent
- HLA-matched donor able to donate
- Prior allogeneic HSCT
- Other active malignancy
- Active or uncontrolled infection
- Active/symptoms of central nervous system (CNS) disease
- Pregnancy or lactation
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description NiCord® (omidubicel) NiCord® (omidubicel) NiCord® is a cryopreserved stem/progenitor cell based product comprised of: 1. ex vivo expanded, umbilical cord blood-derived hematopoietic CD34+ progenitor cells (NiCord® cultured fraction (CF)) 2. the non-cultured cell fraction of the same Cord Blood Unit (CBU) (NiCord® Non-cultured Fraction (NF)) consisting of mature myeloid and lymphoid cells. Both fractions, i.e. NiCord® CF and NiCord® NF, will be kept frozen until they are thawed and infused on the day of transplantation. Unmanipulated CBU(s) Cord Blood Unit -
- Primary Outcome Measures
Name Time Method Time to Neutrophil Engraftment post-transplant up to 42 days The time to engraftment of neutrophils \>500/μl was defined as per Center for International Blood and Marrow Transplant Research (CIBMTR) standards, requiring donor chimerism for neutrophil engraftment.
- Secondary Outcome Measures
Name Time Method Days Alive and Out of Hospital in the First 100 Days Post-transplantation 100 days post-transplantation Days alive and out of hospital in the first 100 Days post-transplantation for the Intent to Treat Population
Number of Participants With Platelet Engraftment by 42 Days Post-transplantation 42 days post-transplantation Platelet engraftment defined as the first day of a minimum of three consecutive measurements on different days such that the patient has achieved a platelet count \> 20 × 10\^9/L with no platelet transfusions during the preceding seven days (counting day of engraftment as one of the preceding seven days) for the Intent to Treat Population
First Grade 2/3 Bacterial or Invasive Fungal Infections by 100 Days Following Transplantation 100 days post-transplant First Bacterial Infection Grades 2-3 or Invasive Fungal Infection by 100 Days following Transplantation for the Intent to Treat Population
Trial Locations
- Locations (52)
University of Kansas Cancer Center
🇺🇸Westwood, Kansas, United States
UCLA
🇺🇸Los Angeles, California, United States
Robert Debré
🇫🇷Paris, France
Loyola University, Cardinal Bernardin Cancer Center
🇺🇸Maywood, Illinois, United States
Rutgers Cancer Institute of New Jersey
🇺🇸New Brunswick, New Jersey, United States
Hadassah Medical Center
🇮🇱Jerusalem, Israel
Hospital Universitario Virgen del Rocío
🇪🇸Sevilla, Spain
National University Cancer Institute
🇸🇬Singapore, Singapore
Tel-Aviv Sourasky Medical Center
🇮🇱Tel Aviv, Israel
Ospedale Pediatrico Bambino Gesù
🇮🇹Rome, Italy
Chaim Sheba Medical Center, The Edmond and Lily Safra Children's hospital
🇮🇱Tel HaShomer, Israel
Rambam
🇮🇱Haifa, Israel
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo
🇧🇷São Paulo, Brazil
Hospital Israelita Albert Einstein
🇧🇷São Paulo, Brazil
Children's Medical Center of Dallas
🇺🇸Dallas, Texas, United States
Prinses Maxima Centrum voor Kinderoncologie B.V.
🇳🇱Utrecht, Netherlands
ICO Bellvitge
🇪🇸Barcelona, Spain
Careggi University Hospital
🇮🇹Florence, Italy
The Royal Marsden NHS Foundation Trust
🇬🇧Sutton, Surrey, United Kingdom
Stanford University Cancer Institute
🇺🇸Palo Alto, California, United States
Northwestern University
🇺🇸Evanston, Illinois, United States
The University of Maryland Medicine Center
🇺🇸Baltimore, Maryland, United States
West Cancer Clinic
🇺🇸Germantown, Tennessee, United States
University of Virginia Cancer Center
🇺🇸Charlottesville, Virginia, United States
Instituto Nacional de Câncer José Alencar Gomes da Silva - INCA
🇧🇷Rio De Janeiro, Brazil
Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo
🇧🇷São Paulo, Brazil
Rabin Medical Center, Beilinson Hospital
🇮🇱Petach Tikva, Israel
Dana-Dwek Children's Hospital, Tel-Aviv Sourasky Medical Center
🇮🇱Tel Aviv, Israel
Instituto Português de Oncologia de Lisboa Francisco Gentil
🇵🇹Lisbon, Portugal
Singapore General Hospital
🇸🇬Singapore, Singapore
Hospital Universitari Vall d'Hebron pediatrics
🇪🇸Barcelona, Spain
University Hospital Vall d'Hebron
🇪🇸Barcelona, Spain
Sant Joan de Deu
🇪🇸Barcelona, Spain
Hospital de la Santa Creu i Sant Pau
🇪🇸Barcelona, Spain
Hospital Universitario La Fe
🇪🇸Valencia, Spain
Hospital Universitario y Politécnico La Fe (pediatric)
🇪🇸Valencia, Spain
Queen Elizabeth Hospital
🇬🇧Birmingham, United Kingdom
St James Hospital
🇬🇧Leeds, United Kingdom
Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo Pediatrics
🇧🇷São Paulo, Brazil
Manchester University NHS Foundation Trust
🇬🇧Manchester, United Kingdom
City of Hope
🇺🇸Los Angeles, California, United States
UC San Diego Moores Cancer Center
🇺🇸San Diego, California, United States
Children's Hospital Colorado
🇺🇸Denver, Colorado, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
University of Minnesota Masonic Cancer Center
🇺🇸Minneapolis, Minnesota, United States
Dana-Farber Cancer Institute
🇺🇸Boston, Massachusetts, United States
Duke University Medical Center
🇺🇸Durham, North Carolina, United States
Henry Ford Medical Center
🇺🇸Detroit, Michigan, United States
Cleveland Clinic Children's
🇺🇸Cleveland, Ohio, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
University Medical Center Utrecht
🇳🇱Utrecht, Netherlands
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